Access to Medicines Prior to Authorisation

Under European and Irish legislation, medicinal products (medicines) must be authorised before being marketed. In Ireland, there are two exemptions from authorisation that are relevant to patients for the treatment of conditions where there are medical needs that cannot be met by authorised medicines. These are:

Supply through participation in an approved clinical trial; or

In accordance with the specifications of a practitioner for use by his individual patients on his direct personal responsibility, in order to fulfil the special needs of those patients.

Currently, there is no provision in Irish legislation for the approval of compassionate use programmes for specific groups of patients with an unmet medical need.

Clinical Trials

Medicines intended for use in authorised clinical trials are exempt from the requirement for authorisation but do need to fulfil the requirements outlined in the European Communities (Clinical Trials on Medicinal Products for Human Use) Regulations 2004 (SI No 190 of 2004), as amended.

In general, medicines that have not been authorised are first made available to patients through clinical trials, and doctors can advise their patients as to whether there is a suitable clinical trial that they can enter. Information on trials being conducted in Ireland is also available from the EU Clinical Trials Register.

Another option is for a patient to enter an ‘expanded access programme’ whereby a company makes an unauthorised medicine available to patients who can benefit from it. For example, patients who have been treated with the medicine during a clinical trial and wish to continue treatment may be able to do so via an expanded access programme.

There is no legislative provision for the authorisation of expanded access programmes. If the programme fulfils the definition of a clinical trial, it is required to be authorised under that legislation. In the event that a programme does not fulfil the definition of a clinical trial, supply of a medicine under the programme falls within the exemption relating to supply in accordance with the specifications of a practitioner for use by his individual patients on his direct personal responsibility (see below).

Supply of unauthorised medicines in accordance with the specifications of a practitioner for use by his individual patients

Irish legislation recognises the possibility for registered doctors and dentists to access unauthorised medicines for patients under their care. This is addressed in the Medicinal Products (Control of Placing on the Market) Regulations, 2007, (SI No 540 of 2007) as amended. An unauthorised medicine is considered ‘exempt’ from authorisation when it is supplied to the order/prescription of a registered doctor or dentist for use by his individual patients on his direct responsibility in order to fulfil the special needs of those patients.

The HPRA does not issue approvals for use of exempt medicines as they have not been assessed against the criteria of safety, quality and efficacy. Wholesalers and manufacturers based in Ireland are required to notify the HPRA when they are importing exempt medicines for the purposes of supply in Ireland. This permits the HPRA to track availability of exempt medicines and to assist in a recall if this is necessary. The wholesaler or manufacturer is required to have processes in place to capture and record any adverse reaction notified in relation to an exempt medicine and to report this to the HPRA.

The HPRA does not keep records of patients that are being treated with exempt medicines. In comparison, retention of patient information is generally a requirement of an authorised compassionate use programme.